Wellcome Trust’s Syncona Bets On Gene Therapy in Eye Disease
This article was originally published in The Pink Sheet Daily
Executive Summary
The profit-oriented evergreen fund of Britain’s charitable foundation The Wellcome Trust, Syncona, has invested £12 million in an academic spin-out to develop and commercialize therapies for treating degenerative eye diseases.
A novel gene therapy that has restored some sight in people born with an inherited, progressive form of blindness is the cornerstone of a new company established with funding from Syncona Partners LLP, the investment arm of The Wellcome Trust.
Syncona is injecting £12 million ($20 million) into NightstaRx Ltd (“Nightstar”), a spin-out from the University of Oxford and its research commercialization company Oxford University Innovation Ltd. Nightstar aims to treat an inherited form of progressive blindness called choroideremia. Developed by Robert MacLaren, a professor at Oxford’s Nuffield Laboratory of Ophthalmology, the gene therapy has shown very promising results in a clinical trial published in The Lancet in mid-January. Nightstar has since bought an exclusive license from Isis Innovation to develop the medicine.
Convincing Evidence
The gene therapy uses a small modified virus, AAV.REP1, to deliver the correct version of the mutated gene that causes choroideremia to cells in the retina of the eye. Six months after treatment with this therapy, the first six patients showed improvement in their vision in dim light and two of the six were able to read more lines on the eye chart, according to the Jan. 16 Lancet paper.
The vector is currently in Phase I trials and follow-on tests are expected to begin in 2016.
But Syncona said the therapy’s promise was clear, prompting it to move quickly.
“We’re not waiting for completion of those trials before starting the next phase of trials because we’ve seen enough in the data published in the Lancet to make us confident about this drug,” said Chris Hollowood, a partner at Syncona and Nightstar’s chairman. So the company is planning a Phase III trial it hopes will start soon, “and then we’ll start what we hope to be a pivotal trial,” he said in an interview.
MacLaren will now advise the company and sit on the board of directors. The other two researchers involved in discovering the therapy are Miguel Seabra of Imperial College London and Matthew During, a professor of Virology at Ohio State University.
Choroideremia is caused by a mutation in the CHM gene on the X chromosome and affects an estimated 1 in every 50,000 people. The condition causes progressive loss of vision due to degeneration of the choroid, retinal pigment epithelium, and retina. There is no treatment for choroideremia and eventually the photoreceptor cells – the rods and cones in the retina that respond to light by sending signals to the visual processing areas of the brain – also degenerate, leading to complete blindness by middle age.
It’s the second start-up investment made from Syncona’s maiden £200 million ($331 million) fund since its formal inception a year ago (Also see "Wellcome Trust Launches £200 million “Evergreen” Investment Fund" - Pink Sheet, 2 Jan, 2013.). Syncona previously took a small stake in Cambridge Epigenetix Ltd. (CEGX), a bioscience tools company.
“While that investment was undisclosed it was relatively modest,” Hollowood said, declining to elaborate.
Newly established Nightstar aims to use AAV.REP1 as its foundation for building a profitable pipeline of therapies for treating retinal dystrophies.
“This is the asset that allows us to get going and build a team and we have already been working on the pipeline and will announce it when we’re ready,” Hollowood said.
“Because choroideremia is a rare and severe disease we can give it the capital it requires all the way to market,” said Hollowood, which could take the company through to profitability and obviate the need to find a buyer for the company to ensure the investors’ return.
He added though that Nightstar wouldn’t say “no” to an attractive buy out later on.
“We are a financially-driven investor and if someone turns up half way through the process and gives us an attractive offer we’ll obviously listen to it,” Hollowood said.
He said gene therapy is of particular interest for the evergreen fund.
“By definition it can be targeted to deliver high efficacy and you need to do that, both to get patients and to get reimbursements in the future.”